Genomic and Biologic Therapies for ADRD (U01 Clinical Trial Optional)
National Institutes of Health
Summary
This Notice of Funding Opportunity (NOFO) supports the translation of genome editing and other biologic therapies for Alzheimer's Disease-Related Dementias (ADRD). The program spans the full translational pipeline, from therapeutic lead optimization and drug product development through IND-enabling studies and early clinical evaluation. Supported activities may include optimization of editing constructs and delivery systems; assessment of bioactivity, target engagement, durability, pharmacokinetics, and bioavailability; demonstration of in vivo efficacy; execution of safety pharmacology and toxicology studies; and development of scalable manufacturing processes, formulation strategies, and quality attributes aligned with regulatory expectations. ADRD include Frontotemporal Dementia (FTD); Lewy Body Dementias (LBD), including Dementia with Lewy Bodies (DLB) and Parkinson's Disease Dementia (PDD); Vascular Contributions to Cognitive Impairment and Dementia (VCID); and Multiple Etiology Dementias (MED).
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Eligibility
Other Eligible ApplicantsEligible Agencies of the Federal Government;U.S. Territory or Possession;Faith-based or Community-based Organizations;Regional Organizations;Non-domestic (non-U.S.) Entities (Foreign Institutions). | applicant types: Special district governments; Small businesses; County governments; Public housing authorities/Indian housing authorities; Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education; Native American tribal governments (Federally recognized); State governments; City or township governments; Others (see text field entitled "Additional Information on Eligibility" for clarification); For profit organizations other than small businesses; Public and State controlled institutions of higher education; Independent school districts; Native American tribal organizations (other than Federally recognized tribal governments); Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education; Private institutions of higher education